Walking, running, and even swimming were once said to be 'impossible' for 4 year old Rylae-Ann Poulin by doctors, but now, thanks to pioneering gene therapy, Rylae is living a 'completely different life.' 👏
When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age.
This photo provided by Judy Wei shows her daughter, Rylae-Ann Poulin, the first time she was admitted to an ICU, at a Singapore hospital in September 2018 At around 3 months old, Rylae-Ann began having spells her parents thought were seizures — her eyes would roll back and her muscles would tense. Fluid sometimes got into her lungs after feedings, sending her to the emergency room. Doctors thought she might have epilepsy or cerebral palsy.
‘It gets put into the brain cells and then the brain cells make the dopamine,’ said Stuart Peltz, CEO of PTC Therapeutics. Bankiewicz said all 40 or so patients in his team’s NIH-funded study also saw significant improvements. His surgical approach is more involved and delivers the treatment to a different part of the brain. It targets relevant circuits in the brain, Bankiewicz said, like planting seeds that cause ivy to sprout and spread.
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